Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants

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Grant Award Details

Grant Number:
DISC0-18038
Investigator(s):
Institution:
Type:
PI

Award Value:
$4,606,248
Status:
Pre-Active

Grant Application Details

Application Title:

Base Editing, Single-Cell Multiomics, and Cardiac Organoids to Decode Genetic Variants

Public Abstract:
Research Objective

Develop a high-throughput platform combining iPSC-derived cardiac organoids and CRISPR base editing to functionally assess missense variants in hypertrophic cardiomyopathy.

Impact

(i) Limited tools for functional interpretation of missense variants, (ii) lack of scalable human cardiac models, and (iii) poor understanding of variant effects across genetic backgrounds.